It's a new year, which means it's a new opportunity for drugmakers hoping to pass FDA muster for new therapies––some of which could be sniffing after blockbuster sales.
In the first quarter, the FDA is set to decide on a group of new drugs that come with high hopes, high scrutiny and, in some cases, raised eyebrows from the agency staff who have reviewed them.
Here are the FDA finish lines we'll be watching most closely over the next three months.
Drug: Tazemetostat
Indication: Advanced epithelioid sarcoma
Company: Epizyme
PDUFA date: Jan. 23
Despite facing some scathing criticism from FDA staff, Epizyme's sarcoma fighter tazemetostat sailed through an advisory committee session with a unanimous vote in December on its way to an approval decision date Jan. 23.
Epizyme is seeking approval for tazemetostat in patients with advanced epithelioid sarcoma who can’t undergo surgery to remove their tumors. It's a rare cancer of the soft tissue that can spread to other areas. It's also difficult to diagnose, so it may not be discovered until its later stages. The drug’s application is based on data from about 100 patients who made up two groups—cohorts 5 and 6—in a larger study that tested tazemetostat in various tumor types.
In terms of peak sales, the indication is small––Jefferies analyst Michael Yee put the U.S. market at $50 million––but it would get Epizyme's foot in the door. The company's also seeking an approval for tazemetostat in follicular lyphoma, which has a market closer to $500 million.
Drug: Bempedoic Acid
Indication: High LDL cholesterol
Company: Esperion
PDUFA date: Feb. 21
There's a lot on the line for Esperion with its cholesterol-fighter bempedoic acid after the drugmaker has withstood a dwindling share price and faces a staggered loan tied to the med's approval decision Feb. 21.
Esperion faces an FDA decision on bempedoic acid as a solo treatment for high cholesterol and as a combo treatment with ezetimibe, a Merck & Co. cholesterol-fighter marketed as Zetia.
In a yearlong phase 3 trial released in May 2018, bempedoic acid showed it can lower LDL cholesterol levels by an additional 20% over 12 weeks in high-risk patients taking statins. However, the study also reported 13 deaths in its treatment arm, a rate of 0.9%, about three times that seen with placebo.
On a later conference call with investors, Esperion’s senior vice president for clinical research, Bill Sasiela, said the deaths stemmed from an older, less-healthy study population, two-thirds of which were current or former smokers.
In June, Esperion struck a $200 million funding agreement for a staggered loan, which granted Esperion $125 million up front with up to $75 million to follow as bempedoic acid wins FDA approval and comes to market. Over the last six months, Esperion's shares have dropped around 2.45% to $59.84.
Drug: Teprotumumab
Indication: Thyroid eye disease
Company: Horizon Therapeutics
PDUFA date: March 8
Horizon Therapeutics is hoping to pivot away from its reputation of repurposing older meds at astronomical prices into rare diseases, and the approval of eye drug teprotumumab could help it get over the hump.
The treatment for thyroid eye disease (TED) received an FDA advisory committee's unanimous backing in December ahead of its March 8 decision date. Teprotumumab inhibitors the insulin-like growth factor type 1 receptor for TED, a rare autoimmune disorder that can lead to blurred or double vision. Patients typically seek treatment when they notice their eyes bulging in photos or that their eyes and eyelids constantly seem to be red and swollen, Raymond Douglas, M.D., Ph.D., one of the leaders of teprotumumab’s phase 3 study, said in a previous interview.
If approved, teprotumumab would be the first treatment for TED that changes the course of the disease rather than just addressing symptoms. Today, patients take steroids to keep swelling at bay or undergo bone-shaving surgery that returns the eyeball to a normal position i...
(All Rights Reserved)